Regenxbio Inc.’s gene therapy in treating Duchenne muscular therapy produced positive initial phase I/II results from its first five patients. However, the company’s stock (NASDAQ:RGNX) had shuddered at midday as shares were going for $8.46 each, a drop of 16% since the market opened. The new study data for RGX-202 showed an improved performance of an average of 4 points from baseline and 4.8 points compared to patients’ natural history on the North Star Ambulatory Assessment and improvement on two timed function tests. Those patients were ages approximately 6 to 12 years old when receiving the treatment at the second dose level.

Ascletis’ denifanstat meets phase III endpoints in acne

Ascletis Pharma Inc.’s once-daily oral fatty acid synthase inhibitor, denifanstat, demonstrated statistically significant and clinically meaningful improvements compared to placebo, meeting all primary and secondary endpoints in a phase III trial for moderate to severe acne vulgaris. Conducted in China, the randomized, double-blind, placebo-controlled, multicenter trial evaluated 50-mg denifanstat once daily or matching placebo. At week 12, treatment success was 33.2% compared to 14.6% for placebo, and the reduction from baseline in total lesion count was 57.4% in the treatment arm compared to 35.4% for placebo.

EMA: New clinical guideline includes pregnant, breastfeeding patients

The EMA has issued a new guideline on how to include and/or retain pregnant and breastfeeding women in clinical trials, in a move that it says “marks a change in the paradigm.” The aim is to ensure that trial sponsors generate robust clinical data in these populations. That could lead to a significant shift in recruitment, given that currently fewer than 0.4% of clinical trials registered in the EMA’s trials database include women who are pregnant, and this falls to 0.1% in those who are breastfeeding.

Regulatory, financial hurdles the gap between science and access

Children with solid tumors who relapse are being treated with the same chemotherapy they would have been given 40 years ago, as “there have been no major approvals for pediatric solid tumors,” Catherine Bollard, senior vice president and chief research officer at Children’s National Hospital, said today at an FDA roundtable on cell and gene therapies (CGTs). The problem isn’t the science. She noted that many groups are working on curative CGTs “for these children who have lost all other hope for survival,” adding that the real gap is that “big pharma doesn’t see the business model because it’s a rare disease.” Given the regulatory and reimbursement hurdles, CGTs often are not profitable, if even sustainable, several speakers said. Designed as a listening session, the roundtable discussion raised several issues the FDA, CMS and NIH will need to address to make scientific advances available to the people who need them.

Taurine aging biomarker story gets more complicated

A publication based on longitudinal and cross sectional data and led by researchers at the U.S. NIH’s National Institute on Aging published on June 5, 2025, in Science has stated that the impact of taurine supplementation at delaying aging or treating aging-related conditions in context-dependent, and that the circulating levels of taurine are impacted by factors unique to each individual rather than declining with age. To qualify taurine as a true marker of aging, it should change with age across diverse populations over time and ideally supported by longitudinal data.

Signify Bio’s $15M round advances in situ protein therapeutics

Turning the human body into a biofactory of precision protein therapeutics is the focus of newly launched Signify Bio, which emerged with an oversubscribed $15 million initial financing to advance three platforms with broad potential across therapeutic areas. The company’s origins began in a laboratory run by Daniel Siegwart at the University of Texas Southwestern Medical Center and was ignited by a friendship between Siegwart and Signify Bio’s co-founder and CEO, RA Session II. “Dan and I are really close friends. We’ve been working together close to 10 years now,” Session told BioWorld.

Ligachem inks two mystery ADC deals with CSPC subsidiary Novarock

Ligachem Biosciences Inc. signed two license agreements with Novarock Biotherapeutics Inc., a subsidiary of CSPC Pharmaceutical Group Ltd., to bring in two of Novarock’s antibodies and create antibody-drug conjugate (ADC) candidates with novel cancer targets. Details on the counterparty, financial terms, assets and indications were scant; however, Ligachem’s deal announced June 5 matches those hinted at by CSPC on the Hong Kong Stock Exchange May 30, which alluded to three potential deals worth $5 billion combined.

Also in the news

Alvotech, Anaptysbio, Ascletis, Atyr, Bayer, Camurus, Cellectar, Commit, Corvus, Elicio, Eli Lilly, Halo, Hyloris, Immix, Innovent, Insilico, Janssen, Johnson & Johnson, Legend, Merus, Moleculin, Oncovita, Repertoire, Roche, Sarepta, SFL Pharmaceuticals Deutschland, Sionna, Takeda, Tolremo, Trevi, Vasa, Vividion, Xbrane